Amyotrophic lateral sclerosis (ALS) is a neurodegenerative, life-threatening disease characterized by progressive muscular paralysis reflecting degeneration of motor neurons. While the average survival time is 3 years, about 10 percent of people with ALS will survive 10 years.
Preclinical studies show that masitinib is capable of exerting neuroprotection in both the central and peripheral nervous systems by inhibiting the functionality of different cells implicated in ALS progression. AB Science has completed a phase 2/3 human clinical trial with promising results in a population accounting for about 85% of ALS patients. A confirmatory phase 3 study will be launched in 2019.
In recognition of the critical need for new treatments, masitinib received orphan drug designation for ALS from both the European Medicine Agency (EMA) and the U.S. Food and Drug Administration (FDA).
Summary of clinical program
|Phase||Design||Population||Primary Target||Patient Target||IDMC recommandation||Study Status||Related publications|
|2/3||Prospective, double-blind, placebo-controlled, parallel groups study||Patients suffering from Amyotrophic Lateral Sclerosis||Change from baseline to week 48 in Amyotrophic Lateral Sclerosis functional rating scale (ALSFRS)-Revised||400||NA||Study completed||–|
Phase 2/3 study
- 27% slowing of ALSFRS-R deterioration at week 48 (primary analysis)
- 29% slowing of deterioration in quality-of-life (ALSAQ-40)
- 22% slowing of deterioration in respiratory function (FVC)
- 25% delay in disease progression (survival-to-event analysis)
Trias E, King PH, Si Y, Kwon Y, Varela V, Ibarburu S, Kovacs M, Moura IC, Beckman JS, Hermine O, Barbeito L.
JCI Insight. 2018 Oct 4;3(19). pii: 123249. doi: 10.1172/jci.insight.123249.
Evidence for mast cells contributing to neuromuscular pathology in an inherited model of ALS.
Trias E, Ibarburu S, Barreto-Núñez R, Varela V, Moura IC, Dubreuil P, Hermine O, Beckman JS, Barbeito L.
JCI Insight. 2017 Oct 19;2(20). pii: 95934. doi: 10.1172/jci.insight.95934.
Post-paralysis tyrosine kinase inhibition with masitinib abrogates neuroinflammation and slows disease progression in inherited amyotrophic lateral sclerosis.
Trias E, Ibarburu S, Barreto-Núñez R, Babdor J, Maciel TT, Guillo M, Gros L, Dubreuil P, Díaz-Amarilla P, Cassina P, Martínez-Palma L, Moura IC, Beckman JS, Hermine O, Barbeito L.
J Neuroinflammation. 2016 Jul 11;13(1):177. doi: 10.1186/s12974-016-0620-9.
ALS Clinical Trials Review: 20 Years of Failure. Are We Any Closer to Registering a New Treatment?
Petrov D, Mansfield C, Moussy A, Hermine O.
Front Aging Neurosci. 2017 Mar 22;9:68. doi: 10.3389/fnagi.2017.00068. eCollection 2017. Review.
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